This page shows the latest beta thalassaemia news and features for those working in and with pharma, biotech and healthcare.
bluebird bio will continue to focus on delivering gene therapies in its core areas – beta-thalassaemia, cerebral adrenoleukodystrophy and sickle cell disease. ... This includes its beta-thalassaemia gene therapy betibeglogene autotemcel, which is
bluebird bio’s beta thalassaemia gene therapy Zynteglo scores promising long-term data. ... bluebird bio also posted long-term data for its own beta thalassaemia gene therapy Zynteglo (betibeglogene autotemcel) at ASH 2020.
in patients with transfusion-dependent beta thalassaemia (TDT).
LentiGlobin is approved under the brand name Zynteglo in Europe for the treatment of dependent beta thalassaemia (TDT) who do not have a matching donor for a stem cell transplant.
It is also working on its own in-house projects, namely SLN124 for beta thalassaemia, which is in phase 1b testing, and SLN360 which targets Lp(a) and is in early
Other developers are aiming to produce therapies which avoid the causes of GVHD – that includes bluebird bio, whose beta thalassaemia treatment Zynteglo/LentiGlobin is designed to avoid the complications of GVHD
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An in-depth look at beta thalassaemia for Rare Disease Day. Beta thalassaemia major is a serious, lifelong genetic blood disorder. ... At least 75, 000 children are born. with thalassaemia worldwide every year, of which 10, 000 have the debilitating form
treatment ozanimod and myelodysplastic syndromes/beta thalassaemia candidate luspatercept – to deliver on their potential.
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